4 completed, 1 terminated, 1 unknown. 4 Phase 2, 3 Phase 1, 1 Phase 3.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 4 | Completed 2, Terminated 1, Unknown 1 |
| Phase 1 | 3 | Completed 2, Terminated 1 |
| Phase 3 | 1 | Unknown 1 |
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Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| A Safety and Efficacy Study of LYS-GM101 Gene Therapy in Patients With GM1 Gangliosidosis LYS-GM101 - GM1 Gangliosidosis | Phase 1 / Phase 2 | Terminated | LYSOGENE | 5 | 2021-05-11 | 2023-06-09 |
| Interviews and Video Capture in Patients With GM1 Gangliosidosis Natural history - GM1 Gangliosidosis | Completed | LYSOGENE | 25 | 2020-04-20 | 2023-06-08 | |
| Study of AAVrh10-h.SGSH Gene Therapy in Patients With Mucopolysaccharidosis Type IIIA (MPS IIIA) LYS-SAF302 - Mucopolysaccharidosis Type IIIA | Phase 2 / Phase 3 | Unknown | LYSOGENE | 20 | 2018-12-17 | 2021-08-31 |
| Natural History Study of Patients With MPS IIIA Mucopolysaccharidosis IIIA | Completed | LYSOGENE | 23 | 2016-04-01 | 2021-08-31 | |
| Long-term Follow-up of Sanfilippo Type A Patients Treated by Intracerebral SAF-301 Gene Therapy SAF-301 - Mucopolysaccharidosis Type III A, Sanfilippo Disease Type A | Phase 1 / Phase 2 | Completed | LYSOGENE | 4 | 2013-05-01 | 2017-06-20 |
| Intracerebral Gene Therapy for Sanfilippo Type A Syndrome SAF-301 - Mucopolysaccharidosis Type III A, Sanfilippo Disease Type A | Phase 1 / Phase 2 | Completed | LYSOGENE | 4 | 2011-08-01 | 2014-05-06 |
Lysosomal Storage Diseases (3) · Mucopolysaccharidosis III (3) · Gangliosidosis, GM1 (2) · Genetic Diseases, Inborn (2) · Metabolic Diseases (2) · Neurodegenerative Diseases (2) · Nervous System Diseases (1)
France (5) · United Kingdom (3) · United States (3) · Germany (2) · Netherlands (2) · Brazil (1)